Last updated on October 2018

Study of the Effect of Velaglucerase Alfa (VPRIV ) on Bone-related Pathology in Treatment-na ve Participants With Type 1 Gaucher Disease


Brief description of study

The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by DXA after 24 months of treatment.

Clinical Study Identifier: NCT02574286

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Kaiser Permanente
Los Angeles, CA United States
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Priya Kishnani, MD

Duke University Medical Center
Durham, NC United States
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Can Ficicioglu

The Children's Hospital of Philadelphia
Philadelphia, PA United States
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Jesus Villarrubia Espinosa, MD, PhD

Hospital Universitario Ramon y Cajal
Madrid, Spain
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Pilar Giraldo, MD

Hospital Quironsalud Zaragoza
Zaragoza, Spain
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Patrick B Deegan, MD

Addenbrooke's Hospital
Cambridge, United Kingdom
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Derralynn A Hughes, MD

Royal Free Hospital
London, United Kingdom
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Joel Charrow, MD

Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, IL United States
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Cedars Sinai Medical Center
Beverly Hills, CA United States
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Jaime Vengoechea Barrios, MD

Emory Genetics
Atlanta, GA United States
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Heather Lau, MD

NYU School of Medicine
New York, NY United States
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Hagit Baris Feldman

Rambam Health Care Campus
Haifa, Israel
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Ari Zimmerman, MD

Shaare Zedek Medical Center
Jerusalem, Israel
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Noa Ruhrman Shahar

Rabin Medical Center
Petah Tikva, Israel
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